Gene Therapy and Vision Restoration

What is the underlying cause of Molly's visual impairment?

• A mutation in a gene responsible for a specific eye protein.

• Damage to the optic nerve during birth.

• A viral infection that affected her vision.

• Lack of proper nutrition during early development.

What is the fundamental principle behind gene therapy for hereditary diseases?

• Introducing a mutated gene to trigger an immune response.

• Providing cells with a corrected copy of a mutated gene to produce a functional protein.

• Removing all genes from a cell to prevent disease progression.

• Using medication to suppress the effects of defective proteins.

What is the primary role of the retina in the human eye?

• To regulate the amount of light entering the eye.

• To convert light into neural signals that the brain can interpret.

• To protect the eye from external damage.

• To produce tears for lubrication.

Why are eyes considered a particularly suitable target for gene therapy?

• They are the largest organs in the human body.

• They are easily accessible for treatment and allow for one eye to serve as a control.

• They have a very high rate of cell regeneration.

• They are not connected to the central nervous system.

How do modified viruses deliver corrective genes in gene therapy?

• They replicate within cells, replacing faulty genes.

• They invade cells and introduce the corrective gene without replicating.

• They directly repair mutated genes in the cell nucleus.

• They stimulate the immune system to produce corrective proteins.

Why were Briard dogs considered a suitable animal model for studying Leber congenital amaurosis (LCA)?

• They are easy to breed and maintain in a laboratory setting.

• Their eyes are similar in size to human eyes, and they suffer from a similar genetic blindness.

• They have a naturally strong immune system that can fight off viral vectors.

• They exhibit a rapid progression of blindness, allowing for quick study results.

What is the primary genetic cause of Leber congenital amaurosis (LCA) as described in the video?

• A mutation in the RPE65 gene leading to non-functional photoreceptors.

• An autoimmune response that attacks the retina.

• A viral infection that damages the optic nerve.

• Environmental factors that cause retinal detachment.

What was the primary reason for Jesse Gelsinger's death during a gene therapy trial?

• The corrective gene caused a new, unknown disease.

• His immune system had an overly strong reaction to the viral vector.

• The viral vector replicated uncontrollably, destroying his liver.

• He received an incorrect dosage of the gene therapy.

What was the primary challenge faced by researchers after successfully repeating gene therapy experiments in dogs for blindness?

• Finding enough dogs for further experimentation.

• Adapting the gene therapy for other animal species.

• The ethical and safety concerns surrounding human trials.

• Lack of funding for continued research.

What was Dr. Katherine High's significant contribution to the advancement of gene therapy for blindness?

• She discovered the RPE65 gene responsible for Leber congenital amaurosis.

• She developed a new viral vector that was safer and more effective than previous ones.

• She established a laboratory dedicated to gene therapy and sought out promising research like Bennett's.

• She was the first surgeon to successfully perform gene therapy on a human patient.